19:41 , Mar 8, 2018 |  BC Innovations  |  Emerging Company Profile

Splice time

Skyhawk Therapeutics Inc. is developing small molecules that manipulate the stability of the spliceosome during RNA processing to correct splicing defects that drive disease. Instead of focusing on genes in classic exon-skipping diseases, the newco...
21:40 , Jan 3, 2018 |  BC Innovations  |  Product R&D

Expanding small molecule horizons

With a platform boasting more than a decade of preclinical rigor and $55.3 million in series A funding, Expansion Therapeutics Inc. is the latest company to step into the RNA-binding small molecule arena. The newco...
17:57 , Sep 14, 2017 |  BC Innovations  |  Emerging Company Profile

RNA gets CRISPR

Two of the outstanding hurdles for CRISPR-based gene editing are the safety concerns of permanently changing the cell’s DNA and the efficacy limitations of relying on natural DNA repair mechanisms to fix the cuts. Locana...
07:00 , May 15, 2014 |  BC Innovations  |  Distillery Therapeutics

Indication: Musculoskeletal disease

Indication Target/marker/pathway Summary Licensing status Publication and contact information Musculoskeletal disease Muscular dystrophy Dystrophia myotonica protein kinase (DMPK) In vitro and mouse studies identified a DMPK CUG...
07:00 , Oct 4, 2012 |  BC Innovations  |  Distillery Techniques

Technology: Disease models

Approach Summary Licensing status Publication and contact information Disease models Mouse model for neurological features of myotonic dystrophy type 1 (DM1) A mouse model for DM1 could be useful...
07:00 , Oct 15, 2009 |  BC Innovations  |  Distillery Therapeutics

Indication: Musculoskeletal disease

Indication Target/marker/pathway Summary Licensing status Publication and contact information Musculoskeletal disease Muscular dystrophy Muscleblind-like...
07:00 , Sep 17, 2009 |  BC Innovations  |  Distillery Therapeutics

Indication: Musculoskeletal disease

Indication Target/marker/ pathway Summary Licensing status Publication and contact information Musculoskeletal disease Muscular dystrophy ...
07:00 , Aug 6, 2009 |  BC Innovations  |  Distillery Therapeutics

Indication: Musculoskeletal disease

Indication Target/marker/pathway Summary Licensing status Publication and contact information Musculoskeletal disease Muscular dystrophy Dystrophia...
07:00 , Aug 6, 2009 |  BC Innovations  |  Tools & Techniques

Targeting toxic RNA

Researchers at the University of Rochester have used an antisense molecule to reverse muscular defects in mice with myotonic dystrophy type 1.1 The approach represents a new way to attack the most common form of...
07:00 , Jul 16, 2009 |  BC Innovations  |  Distillery Therapeutics

Indication: Various

Various Muscular dystrophy; ataxia Dystrophia myotonica protein kinase (DMPK); ataxin 3 (ATXN3); muscleblind-like 1 (MBNL1) In vitro studies identified inhibitors of RNA-protein interactions that could help...